Aelis Farma surged over 6% on Tuesday on the Paris Stock Exchange after announcing it has secured regulatory approvals to launch its phase 2B trial targeting Down syndrome.
The biopharmaceutical company reported it has received the green light from the European Medicines Agency (EMA), as well as from relevant authorities and ethics committees in France, Italy, and Spain for the protocol of this clinical study. The trial will focus on AEF0217, one of its two drug candidates, for the treatment of adaptive behavior disorders and cognitive function in individuals with Down syndrome.
The primary objective of the trial is to demonstrate that this specific inhibitor of CB1 receptor signaling in the endocannabinoid system (CB1-SSi), administered once daily over 24 weeks, can lead to improvements in adaptive behaviors compared to a placebo.
Positive results from an earlier phase 1/2 study had previously shown improvements across several key dimensions of adaptive behavior and brain activity.
This new study aims to confirm these benefits on a larger scale and pave the way for the design of phase 3 trials.
The final visit for the last patient is currently anticipated in the first quarter of 2027, with preliminary results expected around mid-2027.
With the company's cash runway extending through early 2028, funding for the trial is secured until results are obtained.
In a statement, Aelis explained that individuals with Down syndrome frequently experience lifelong adaptive behavior and cognitive disorders, for which no pharmacological treatments are currently approved. Their care is limited to educational, behavioral, and supportive interventions, which are considered insufficient to enable these individuals to reach their full potential in terms of autonomy and quality of life.
